About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community.
About the Author
Dongsheng Duan, Ph.D., is the Margaret Proctor Mulligan Professor in Medical Research, at the Department of Molecular Microbiology & Immunology, Neurology, Bioengineering, and Biomedical Sciences at the University of Missouri. He received his medical degree from the West China University of Medical Science, Chengdu, China in 1987 and his Ph.D. degree from the University of Pennsylvania, Philadelphia, USA in 1997. His research in last two decades has focused on the development of adeno-associated virus as a gene therapy vector, pathogenic mechanisms of Duchenne muscular dystrophy, dystrophin biology and preclinical gene therapy for Duchenne muscular dystrophy in murine and canine models.
Jerry R Mendell, M.D. is the Curran-Peters Research Chair and Professor of Pediatrics and Neurology. His career has been devoted to translational clinical science, beginning as a post doctoral fellow at the National Institutes of Health. His life-long focus has been to make a difference in lives burdened with untreatable neuromuscular disease. He has contributed significantly to the muscular dystrophies and spinal muscular atrophy, at first using pharmaceutical tools and since 1999, the time of his first gene therapy trial, he has been dedicated to the successful replacement of mutant genes that undermine the lives of innocent victims.